Table 4.
Examples of gene therapy clinical trials for inherited haematological, ophthalmological and metabolic diseases
| Condition | Target | Clinical trial ID | Method | Delivery | ||
|---|---|---|---|---|---|---|
| Blood | B-thalassemia | BCL11a | NCT03432364 | ZFN | Ex vivo | Non-viral |
| NCT03653247 | Ex vivo | N.R. | ||||
| NCT03655678 | CRISPR-Cas9 | Ex vivo | Non-viral | |||
| NCT03745287 | Ex vivo | Non-viral | ||||
| β-globin | NCT03728322 | CRISPR-Cas9 | iHSC | N.R. | ||
| βA-T87Q-globin | NCT01745120 (Zynteglo) | Gene suppl. | Ex vivo | LV | ||
| Eye | LCA | RPE65 | NCT00643747 | Gene suppl. | In vivo | AAV2 |
| NCT00481546 | In vivo | AAV2 | ||||
| NCT00821340 | In vivo | AAV2 | ||||
| NCT00999609 (Luxturna) | In vivo | AAV2 | ||||
| NCT03872479 | CRISPR-Cas9 | In vivo | AAV5 | |||
| LHON | ND4 | NCT03428178 | Gene suppl | In vivo | AAV2 | |
| NCT03153293 | In vivo | AAV2 | ||||
| NCT02064569 | In vivo | AAV2 | ||||
| NCT02161380 | In vivo | AAV2 | ||||
| Achromatopsia | CNGA3 | NCT02935517 | Gene suppl | In vivo | AAV2 | |
| CNGA3/CNGB3 | NCT03278873 | Gene suppl | In vivo | AAV2/8 | ||
| Choroideremia | REP1 | NCT02341807 | Gene suppl | In vivo | AAV2 | |
| NCT03507686 | In vivo | AAV2 | ||||
| Metabolism | PKD | RPK (Red cell PK) | NCT04105166 | Gene suppl | Ex vivo | LV |
| Haemophilia | F8 | NCT03061201 | Gene suppl | In vivo | AAV2/6 | |
| ADA-SCID | ADA | NCT01380990 (Strimvelis) | Gene suppl | Ex vivo | LV | |
| Fabry disease | GLA | NCT04046224 | Gene suppl | In vivo | AAV2/6 | |
| MPS type I | IDUA | NCT02702115 | ZFN + gene suppl | In vivo | AAV2/6 | |
| NCT03488394 | Gene suppl | Ex vivo | LV | |||
| MPS type II | IDS | NCT03041324 | ZFN + gene suppl | In vivo | AAV2/6 | |
N.R. Not reported. Bold: EMA-approved products